(The) real-world systemic sequential therapy of sorafenib and regorafenib for advanced hepatocellular carcinoma = 진행된 간세포암종에서 소라페닙과 레고라페닙 순차치료의 효과와 안전성 : 실제 임상 환경에서의 국내 다기관 후향적 연구

다국어 초록 (Multilingual Abstract)

Backgrounds/Aim : Regorafenib has been proved as 2nd line systemic therapy for hepatocellular carcinoma (HCC) patients through phase III trial. We analyzed the real-world data to assess clinical efficacy and safety.
Methods : This study was a multicenter, non-comparative, retrospective cohort study. Between July 2017 and May 2019, 133 patients with HCC who received regorafenib after sorafenib therapy were eligible for inclusion in this study. Tumor response was assessed according to modified RECIST criteria. We evaluated time to progression (TTP), progression-free survival (PFS) and overall survival (OS) of regorafenib therapy including prediction factors for prognosis.

Results : Their median age was 60 years, and most patients (82%) were male. Hepatitis B virus infection (68.4%) was the most common etiology of HCC. Most of patients (98.5%) were classified as Child- Pugh A except 2. Eighty-four percent of patients had extrahepatic metastasis and Vascular invasion was presented in 45.1%.
Three patients (2.7%) achieved complete response and 11 (9.8%) patients had a partial response resulting in objective response rate 12.5% in 112 available patients for response assessment. The disease control rate (DCR) was 34.8%.
Median treatment duration of regorafenib was 2.6 months (1.5-4.7 months). During follow up, 38 patients died. On regorafenib, the median OS was 10.0 months (95% CI, 8.4-11.6), the median PFS survival was 2.7 months (95% CI, 2.5- 2.9 months), and the median TTP was 2.6 months (95% CI, 2.4-2.8). The OS rate from the start of regorafenib at 6 months and 1 year were 71.2%, and 38.7%. In multivariate analysis, Child-Pugh score >5 (HR 3.037 95% CI 1.46-6.314; p=0.003), AFP > 400 ng/ml (HR 5.9; 95% CI 2.608-13.349; p< 0.001), and TTP on sorafenib ≥ median (HR 0.441; 95% CI 0.310-0.629; p< 0.001) were independently associated with OS.
In exploratory analysis from the time of sorafenib administration, the median OS from sorafenib administration was 25.8 (95% CI 8.7-42.9 months). The 1 year and 2years survival rate were 79.5% and 53.1%.

Conclusion : Regorafenib was effective in patients with advanced HCC who failed first-line sorafenib in real-life setting, consistent with previous clinical trial. Regorafenib may improve the prognosis of patients who had longer TTP on previous sorafenib therapy.

목차 (Table of Contents)

• I. Abstracts ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 6
• II. Introduction ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 8
• III. Methods ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 11
• IV. Results ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 15
• V. Discussion ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 21
• VI. Conclusion ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 25
• VII. References ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 26
• VIII. Tables and figures ∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙∙ 29